Background: Tropical infectious diseases are called neglected, because they
are, inter alia, characterized by an R&D deficit. A similar deficit exists for rare (orphan) diseases which neither
promise a sufficient return on R&D investment. To encourage the development of treatments for rare diseases, orphan
drug acts were created which contain financial and non-financial incentives for the pharmaceutical industry.
Similar instruments aimed exclusively at neglected diseases do not yet exist. Proposals for a regulatory approach to
promote R&D for neglected diseases include the application of selected orphan drug incentives, or the
implementation of a Medical Research and Development Treaty (MRDT) with national funding obligations for medical R&D. We
compiled and analyzed experts’ opinions on causes for the treatment deficit for neglected diseases and
on desirable and feasible measures to promote neglected disease R&D. Hereby, the focus was on mechanisms contained
in orphan drug regulations and in the Medical Research and Development Treaty draft (Discussion draft 4,
2005). Lastly, we solicited experts’ opinions on the desirability and feasibility of a regulatory instrument to
foster R&D for neglected diseases.

Methods: An international online-Delphi survey was conducted with 117 (first
round) and 56 (second round) experts of different professional backgrounds and professional affiliations who
formulated and ranked causes and solutions related to the treatment deficit for neglected diseases.

Results: In both rounds of survey, the majority of the participating experts
(88.4% first round, 86.8% second round) advocated the development of a regulatory instrument to promote R&D for
neglected diseases. Most experts (77.9% first round, 79.3% second round) also considered this to be a feasible
option. With the exception of market exclusivity, which was viewed critically, key provisions contained in orphan
drug regulations were judged favorably also for neglected diseases. A majority (87.1% first round, 77.2% second round)
supported national funding obligations for neglected diseases which are proposed by the Medical Research
and Development Treaty draft.

Conclusions: While not all features of orphan drug regulations and of the
MRDT draft received equal support, the view was expressed that a regulatory instrument would be a desirable and
feasible measure to promote R&D for neglected diseases.